A first-ever treatment for desmoid tumours is approved for the NHS in England
What happened
For the first time, there is a medicine approved for use on the NHS in England that treats desmoid tumours. The National Institute for Health and Care Excellence (NICE) decides which treatments the NHS should pay for. It has now issued what it calls "final draft guidance" recommending a drug called nirogacestat. Its brand name is Ogsiveo. The decision was reported on 29 and 30 September 2026. The recommendation is for adults in England who have a desmoid tumour that is growing, and who need drug treatment for it. Patients do not have to wait for the paperwork to be finished. Funding is being provided straight away through the Cancer Drugs Fund. That is a pot of NHS money used to get promising cancer treatments to people quickly, while the final guidance is confirmed. Two separate approvals had to line up for this to happen. The Medicines and Healthcare products Regulatory Agency (MHRA) checks whether a medicine is safe and works well enough to be licensed in the UK. It had already licensed nirogacestat for adults with progressing desmoid tumours, making it the first medicine ever licensed in the UK for this condition. NICE then had to decide whether the NHS should fund it. Now both have said yes. Desmoid tumours are rare. Reports of the NICE decision put the number of new cases at roughly 340 to 410 a year. Sarcoma UK has described the condition as affecting about five to six people in every million.
Why this matters
A desmoid tumour is not quite like most cancers people have heard of. The World Health Organization puts them in a middle category, calling them "intermediate" tumours. Here is the good news in that. Desmoid tumours do not usually spread to other parts of the body the way many cancers do. But they can grow into the tissue around them, and that is where the harm comes from. Depending on where a tumour sits, it can press on muscles, nerves or organs. They often appear in early adulthood. They can cause pain, swelling, tiredness and difficulty moving about. They can change how a person looks. Living with one can take a real toll on mental health. Many people need check-ups for the rest of their lives. Until now, the choices were limited. Surgery was often not the answer, because the tumour frequently grew back afterwards. Doctors sometimes turned to other drugs "off-licence" - medicines approved to treat something else, used here because there was nothing better available. That helped some people. But it is not the same as having a treatment that has been tested in this condition and approved for it. Dr Alex Lee, a consultant medical oncologist at The Christie NHS Foundation Trust, said the arrival of nirogacestat "provides clinicians with access to a targeted drug treatment of established effectiveness". He described the decision as "a significant forward step that meets a longstanding unmet need for patients with this challenging condition". Jacqui Black, who founded the charity Desmoid Aid UK, described what these tumours do to the people who have them. They "require life-long monitoring and cause chronic pain, visible disfigurement and restricted mobility".
What the evidence actually says
The approval rests on a study called the DeFi trial, published in the New England Journal of Medicine in 2023. It was a fair test, and worth understanding. A total of 142 adults with growing desmoid tumours took part. Seventy were given nirogacestat, a tablet taken twice a day. Seventy-two were given a placebo - a dummy tablet with no medicine in it. Neither the patients nor their doctors knew who was getting which. The results were clear: - Nirogacestat cut the risk of the tumour growing, or of death, by 71% compared with the placebo. - After two years, 76% of people taking nirogacestat had not had their tumour grow. On placebo, it was 44%. - Tumours actually shrank in 41% of people on nirogacestat, compared with 8% on placebo. In 7% of the nirogacestat group the tumour could no longer be detected at all. That happened in none of the placebo group. - People taking it also reported less pain and a better quality of life. The drug works differently from chemotherapy. It is what scientists call a gamma secretase inhibitor. It blocks a signal inside cells, known as the Notch pathway, that helps these tumours grow. Now the honest limits, which matter just as much: This was a small trial. One hundred and forty-two people is normal for a rare condition, because there simply are not many patients to recruit. But smaller numbers mean less certainty than a trial with thousands of people. The trial compared the drug against a placebo, not against the off-licence medicines doctors were already using. So we cannot say from this study that nirogacestat is better than those older options. We can only say it is clearly better than nothing. It is not for everyone with a desmoid tumour. The recommendation covers adults whose tumour is growing and who need drug treatment. Some desmoid tumours stop growing on their own, or even shrink without treatment. For those people, careful monitoring rather than medication may still be the right approach. Side effects were reported as being in line with what was already known about the drug. Every medicine has them, and a specialist team will go through what to expect.
Qurexa perspective
We should be straight with you about something here. Qurexa delivers prescriptions and supports people in keeping on top of their medicines. But this particular treatment is not one we would ever supply. Specialist cancer medicines like this one are prescribed and dispensed through hospital pharmacy teams, who monitor patients closely. If this treatment is right for you, it will come through your hospital, not through a delivery service. What we do recognise is the shape of the challenge it creates. This is a tablet taken twice a day, for a long time, at home. That is a big shift from treatment that happens in a hospital chair. Taking a medicine twice a day, every day, for years is genuinely hard. It is not a matter of willpower. Life gets busy, routines break, and a missed dose here and there is something almost everyone experiences. In our work supporting people with long-term medicines across Lincolnshire, we see this constantly, and we see how much difference a simple routine makes. If you are starting any long-term treatment, it is worth asking your team what support exists for staying on track. Most hospital teams have clinical nurse specialists who do exactly this, and they would far rather you asked than struggled quietly.
Practical advice
If this news is relevant to you or someone you care for, here is what to do with it. Start with your specialist team, not with your own research. This is a change to what the NHS will fund, not a change to what is right for every individual patient. Your consultant knows your scan results, your symptoms and your history. Do not change or stop any current treatment on your own. If you are on an off-licence medicine that is working for you, that may well still be the best option. Ask, do not act. Useful questions to ask at your next appointment: - Is my tumour currently growing, and would this treatment apply to me? - How does it compare with what I am on now? - What side effects should I watch for, and who do I call if I get them? - Is there anything I should know that could affect future plans, such as starting a family? - Who is my point of contact between appointments? If you are waiting for an appointment, you do not have to wait alone. Sarcoma UK and Desmoid Aid UK both support people living with these conditions, and hearing from others who have been through it helps more than most people expect. Never buy prescription medicines from websites that do not ask for a prescription. Specialist cancer drugs sold this way are not safe, whatever the site claims. And if you already take medicines every day for anything at all, a few small habits go a long way: keep them somewhere you will see them, link them to something you already do daily, and use a pill organiser or a phone alarm if it helps.
What to know
The short version: - NICE has recommended nirogacestat (Ogsiveo) for adults in England with growing desmoid tumours who need drug treatment. This was reported on 29 and 30 September 2026. - It is the first medicine ever licensed and funded in the UK specifically for this condition. - Funding is available immediately through the Cancer Drugs Fund, rather than waiting for final guidance. - In the DeFi trial, it cut the risk of tumours growing by 71% compared with a placebo, and shrank tumours in 41% of patients. - It is not suitable for everyone with a desmoid tumour. Monitoring remains right for some people. - Any decision belongs with your specialist team. Sources: PharmaTimes, "NICE backs first systemic therapy for desmoid tumours", 30 September 2026, https://pharmatimes.com/news/nice-backs-first-systemic-therapy-for-desmoid-tumours/ | PMLiVE, "Ogsiveo recommended by NICE to treat rare soft tissue tumours", 29 September 2026, https://pmlive.com/pharma_news/ogsiveo-recommended-by-nice-to-treat-rare-soft-tissue-tumours | Medscape UK, "UK Approves First Drug Option for Desmoid Tumours", 2026, https://www.medscape.com/viewarticle/uk-approves-first-drug-option-desmoid-tumours-2026a10000qe | Gounder M et al., "Nirogacestat, a gamma-Secretase Inhibitor for Desmoid Tumors", New England Journal of Medicine, 2023, https://www.nejm.org/doi/full/10.1056/NEJMoa2210140 | Sarcoma UK, "Desmoids breakthrough after new drug is approved", 28 November 2023, https://sarcoma.org.uk/news/desmoids-breakthrough-after-new-drug-is-approved/ This article is for general information and does not replace advice from a doctor, pharmacist or other qualified healthcare professional. If you have a desmoid tumour, please speak to your specialist team before making any decision about your treatment.
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